Biologics: Targets & Therapy (Sep 2020)
Gene, Cell and Antibody-Based Therapies for the Treatment of Age-Related Macular Degeneration
Abstract
Engin Akyol, Andrew Lotery Clinical Neurosciences Research Group, Faculty of Medicine, University of Southampton, Southampton General Hospital, Southampton SO16 6YD, UKCorrespondence: Andrew LoteryClinical Neurosciences Research Group, Faculty of Medicine, University of Southampton, Southampton General Hospital, Mailpoint 806, South Lab & Path Block, Tremona Road, Southampton SO16 6YD, UKTel +44 23 8120 5049Email [email protected]: Here we discuss antibody, cell and gene-based therapies that are currently available and under investigation for both wet and dry age-related macular degeneration (AMD). We initially discuss ocular anatomy, AMD modelling as well as the underlying pathophysiology of AMD. The antibody-based trials which have revolutionised the management of wet AMD are reviewed. The latest concepts in antibody therapy for wet AMD such as the port delivery systems, bispecific antibodies, designed ankyrin repeat protein (DARPINs) and brolucizumab are explored. Furthermore, the antibody-based trials targeting the complement pathway to reduce progression of geographic atrophy (GA) in dry AMD are discussed. Stem cell therapy and gene therapy are novel treatment modalities with no established clinical use in wet or dry AMD. Here, we discuss their efficacy so far in clinical trials. Their benefits and risk in the treatment of both wet and dry AMD are evaluated.Keywords: choroidal neovascularisation, geographic atrophy, gene therapy, macular degeneration, stem cell therapy, monoclonal antibodies
